HTT ASO

Unassigned

New Medicines

Huntington's disease

Information

New molecular entity
Roche
Roche

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Antisense oligonucleotide targeting the huntingtin protein, the mutant form of which is neurotoxic
UK prevalence according to GP records has increased from about 5 per 100,000 in 1990 to around 12 per 100,000 in 2010, however incidence has remained constant at about 7 per million person-years: it is thought that this is due to greater willingness to record the diagnosis by GPs, and improved survival [3].
Huntington's disease
Intrathecal